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Fáze 2 základní studie Nab-sirolimu u pacientů s maligními solidními nádory s patogenními změnami v genech TSC1 nebo TSC2 (PŘESNOST 1)

26. října 2026 aktualizováno: Aadi Bioscience, Inc.

Multicentrická otevřená košíková studie fáze 2 s nab-sirolimem pro dospělé a dospívající pacienty s maligními solidními nádory obsahujícími patogenní inaktivační změny v genech TSC1 nebo TSC2

A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes

Přehled studie

Postavení

Dokončeno

Podmínky

Intervence / Léčba

Detailní popis

Study TSC-007 is a prospective phase 2, open-label, multi-institutional basket trial to determine the efficacy and safety profile of nab-sirolimus administered to patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes. Patients will be treated with single agent IV nab-sirolimus until disease progression, or unacceptable toxicity, or until in the opinion of the investigator the patient is no longer benefiting from therapy, or at patient discretion.

Typ studie

Intervenční

Zápis (Aktuální)

128

Fáze

  • Fáze 2

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní místa

  • Jižní Korea
      • Busan, Jižní Korea, 48108
        • Inje University Haeundae Paik Hospital
      • Seoul, Jižní Korea, 03080
        • Seoul National University Hospital
      • Seoul, Jižní Korea, 05505
        • Asan Medical Center
      • Seoul, Jižní Korea, 06351
        • Samsung Medical Center
      • Seoul, Jižní Korea, 03722
        • Severance Hospital, Yonsei University Health System
      • Seoul, Jižní Korea, 08308
        • Korea University Guro Hospital
  • Portoriko
    • Puerto Rico
      • San Juan, Puerto Rico, Portoriko, 00935
        • Pan Oncology Trials, LLC
  • Spojené státy
    • Alabama
      • Birmingham, Alabama, Spojené státy, 35243
        • Alabama Oncology
      • Mobile, Alabama, Spojené státy, 36607
        • Southern Cancer Center
    • Arizona
      • Goodyear, Arizona, Spojené státy, 85395
        • Arizona Oncology Associates
      • Phoenix, Arizona, Spojené státy, 85016
        • Honor Health
      • Prescott Valley, Arizona, Spojené státy, 86301
        • Arizona Oncology Associates
      • Yuma, Arizona, Spojené státy, 85364
        • Yuma Regional Medical Center
    • California
      • Arroyo Grande, California, Spojené státy, 93420
        • PCR Oncology
      • Beverly Hills, California, Spojené státy, 90212
        • Nextgen Oncology
      • Duarte, California, Spojené státy, 91010
        • City of Hope
      • Fullerton, California, Spojené státy, 92835
        • Providence Medical Foundation (Fullerton)
      • Long Beach, California, Spojené státy, 90806
        • MemorialCare
      • Los Angeles, California, Spojené státy, 90033
        • USC Norris Comprehensive Cancer Center
      • Los Angeles, California, Spojené státy, 90095
        • UCLA - Jonsson Comprehensive Cancer Center
      • Napa, California, Spojené státy, 94558
        • Providence Medical Foundation (Napa)
      • Newport Beach, California, Spojené státy, 92663
        • Hoag Memorial Hospital Presbyterian
      • San Diego, California, Spojené státy, 92123
        • Sharp HealthCare
      • San Francisco, California, Spojené státy, 94115
        • UCSF Helen Diller Family Comprehensive Cancer Center
      • Santa Barbara, California, Spojené státy, 93105
        • Ridley-Tree Cancer Center
      • Santa Monica, California, Spojené státy, 90403
        • Sarcoma Oncology Research Center
      • Santa Rosa, California, Spojené státy, 95403
        • Providence Medical Foundation
      • Stanford, California, Spojené státy, 94305
        • Stanford Cancer Center
      • Whittier, California, Spojené státy, 90602
        • The Oncology Institute of Hope & Innovation
    • Colorado
      • Aurora, Colorado, Spojené státy, 80012
        • Rocky Mountain Cancer Centers
      • Boulder, Colorado, Spojené státy, 80303
        • Rocky Mountain Cancer Centers
      • Colorado Springs, Colorado, Spojené státy, 80907
        • Rocky Mountain Cancer Centers
      • Denver, Colorado, Spojené státy, 80220
        • Rocky Mountain Cancer Centers
      • Denver, Colorado, Spojené státy, 80218
        • Rocky Mountain Cancer Centers (Williams St)
      • Lakewood, Colorado, Spojené státy, 80228
        • Rocky Mountain Cancer Centers
      • Littleton, Colorado, Spojené státy, 80120
        • Rocky Mountain Cancer Centers
      • Lone Tree, Colorado, Spojené státy, 80124
        • Rocky Mountain Cancer Centers
      • Longmont, Colorado, Spojené státy, 80501
        • Rocky Mountain Cancer Centers
      • Pueblo, Colorado, Spojené státy, 81008
        • Rocky Mountain Cancer Centers
      • Thornton, Colorado, Spojené státy, 80260
        • Rocky Mountain Cancer Centers
    • Connecticut
      • Hartford, Connecticut, Spojené státy, 06102
        • Hartford Healthcare
      • Norwich, Connecticut, Spojené státy, 06360
        • Eastern Connecticut Hematology and Oncology
    • Florida
      • Altamonte Springs, Florida, Spojené státy, 32701
        • Florida Cancer Specialists - North Division
      • Bonita Springs, Florida, Spojené státy, 34135
        • Florida Cancer Specialists - South Division
      • Bradenton, Florida, Spojené státy, 34205
        • Florida Cancer Specialists - South Division
      • Bradenton, Florida, Spojené státy, 34211
        • Florida Cancer Specialists - South Division
      • Brandon, Florida, Spojené státy, 33511
        • Florida Cancer Specialists - North Division
      • Cape Coral, Florida, Spojené státy, 33909
        • Florida Cancer Specialists - South Division
      • Clearwater, Florida, Spojené státy, 33761
        • Florida Cancer Specialists - North Division
      • Daytona Beach, Florida, Spojené státy, 32117
        • Cancer Specialist - East
      • Fort Lauderdale, Florida, Spojené státy, 33308
        • Holy Cross Hospital
      • Fort Myers, Florida, Spojené státy, 33901
        • Florida Cancer Specialists South Division
      • Fort Myers, Florida, Spojené státy, 33905
        • Florida Cancer Specialists - South
      • Fort Myers, Florida, Spojené státy, 33908
        • Florida Cancer Specialists - South
      • Gainesville, Florida, Spojené státy, 32605
        • Florida Cancer Specialists - North Division
      • Jacksonville, Florida, Spojené státy, 32256
        • Cancer Specialists of North Florida
      • Lakeland, Florida, Spojené státy, 33812
        • TOI Florida
      • Lakeland, Florida, Spojené státy, 33812
        • The Oncology Institute of Hope and Innovation
      • Largo, Florida, Spojené státy, 33770
        • Florida Cancer Specialists - North Division
      • Lecanto, Florida, Spojené státy, 34461
        • Florida Cancer Specialists - North Division
      • Naples, Florida, Spojené státy, 34102
        • Florida Cancer Specialists - South Division
      • Ocala, Florida, Spojené státy, 34474
        • Ocala Oncology
      • Ocala, Florida, Spojené státy, 34474
        • Florida Cancer Specialists - North Division
      • Orange City, Florida, Spojené státy, 32763
        • Florida Cancer Specialists - North Division
      • Orlando, Florida, Spojené státy, 32806
        • Florida Cancer Specialists - North Division
      • Port Charlotte, Florida, Spojené státy, 33980
        • Florida Cancer Specialists - South Division
      • Sarasota, Florida, Spojené státy, 34232
        • Florida Cancer Specialists - South Division
      • Sarasota, Florida, Spojené státy, 34236
        • Florida Cancer Specialists - South Division
      • St. Petersburg, Florida, Spojené státy, 33705
        • Florida Cancer Specialists and Research Institute - North Division
      • St. Petersburg, Florida, Spojené státy, 33707
        • Florida Cancer Specialists - North
      • Stuart, Florida, Spojené státy, 34994
        • Florida Cancer Specialist - East
      • Tampa, Florida, Spojené státy, 33607
        • Florida Cancer Specialists - North Division
      • Tavares, Florida, Spojené státy, 32778
        • Florida Cancer Specialists - North Division
      • Trinity, Florida, Spojené státy, 34655
        • Florida Cancer Specialists - North Division
      • Venice, Florida, Spojené státy, 34285
        • Florida Cancer Specialists - South Division
      • Venice, Florida, Spojené státy, 34292
        • Florida Cancer Specialists - South Division
      • Vero Beach, Florida, Spojené státy, 32960
        • Florida Cancer Specialists - East
      • Wellington, Florida, Spojené státy, 33414
        • Florida Cancer Specialists - East
      • West Palm Beach, Florida, Spojené státy, 33401
        • Florida Cancer Specialists - East
    • Georgia
      • Atlanta, Georgia, Spojené státy, 30303
        • Morehouse School of Medicine
    • Hawaii
      • Honolulu, Hawaii, Spojené státy, 96813
        • Hawaii Cancer Center
    • Illinois
      • Hinsdale, Illinois, Spojené státy, 60521
        • Hope and Healing Cancer Services
      • Rolling Meadows, Illinois, Spojené státy, 60008
        • Northwest Oncology and Hematology
    • Indiana
      • Carmel, Indiana, Spojené státy, 46032
        • Urology of Indiana
      • Fort Wayne, Indiana, Spojené státy, 46804
        • Fort Wayne Medical Oncology and Hematology
      • Goshen, Indiana, Spojené státy, 46526
        • Goshen Health
    • Louisiana
      • Baton Rouge, Louisiana, Spojené státy, 70817
        • Our Lady of the Lake
      • Hammond, Louisiana, Spojené státy, 70403
        • Pontchartrain
    • Maryland
      • Bethesda, Maryland, Spojené státy, 20817
        • American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)
      • Frederick, Maryland, Spojené státy, 21702
        • Frederick Health
      • Rockville, Maryland, Spojené státy, 20850
        • Maryland Oncology Hematology
    • Massachusetts
      • Boston, Massachusetts, Spojené státy, 02215
        • Dana Farber Cancer Institute
      • Fairhaven, Massachusetts, Spojené státy, 02719
        • Southcoast Centers for Cancer Care
    • Michigan
      • Detroit, Michigan, Spojené státy, 48201
        • Barbara Ann Karmanos Cancer Institute
      • Lansing, Michigan, Spojené státy, 48912
        • Sparrow Hospital
    • Minnesota
      • Minneapolis, Minnesota, Spojené státy, 55404
        • Minnesota Oncology Hematology
    • Missouri
      • Bolivar, Missouri, Spojené státy, 65613
        • Central Care Cancer Center
      • Osage Beach, Missouri, Spojené státy, 65065
        • Lake Regional
      • Saint Joseph, Missouri, Spojené státy, 64507
        • Mosaic Life Care
      • Springfield, Missouri, Spojené státy, 65807
        • Oncology Hematology Associates
      • St Louis, Missouri, Spojené státy, 63110
        • Washington University School of Medicine
    • Nebraska
      • Grand Island, Nebraska, Spojené státy, 68803
        • Nebraska Cancer Specialists
      • Omaha, Nebraska, Spojené státy, 68114
        • Nebraska Methodist Hospital
    • Nevada
      • Las Vegas, Nevada, Spojené státy, 89169
        • Comprehensive Cancer Centers of Nevada
      • Las Vegas, Nevada, Spojené státy, 89102
        • OptumCare Cancer Care-Parent
    • New Jersey
      • Belleville, New Jersey, Spojené státy, 07109
        • New Jersey Cancer Care and Blood Disorders
      • Englewood, New Jersey, Spojené státy, 07631
        • Englewood Hospital and Medical Center
      • Florham Park, New Jersey, Spojené státy, 07932
        • Summit Medical Group - NJ
      • Morristown, New Jersey, Spojené státy, 07960
        • Atlantic Health System - Morristown Medical Center
    • New York
      • Buffalo, New York, Spojené státy, 14263
        • Roswell Park Comprehensive Cancer Center
      • East Syracuse, New York, Spojené státy, 13057
        • HOACNY
      • Ithaca, New York, Spojené státy, 14850
        • Cayuga Medical Center
      • New York, New York, Spojené státy, 10065
        • David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center
    • North Carolina
      • Goldsboro, North Carolina, Spojené státy, 27534
        • Southeastern Medical Oncology
    • North Dakota
      • Fargo, North Dakota, Spojené státy, 58102
        • Sanford Health-Fargo
    • Ohio
      • Canton, Ohio, Spojené státy, 44708
        • Aultman Medical Group
      • Cincinnati, Ohio, Spojené státy, 45220
        • TriHealth
      • Cincinnati, Ohio, Spojené státy, 45627
        • University of Cincinnati (UC) - Cancer Institute
      • Cleveland, Ohio, Spojené státy, 44195
        • Cleveland Clinic
      • Toledo, Ohio, Spojené státy, 43623
        • The Toledo Clinic
    • Oklahoma
      • Oklahoma City, Oklahoma, Spojené státy, 73104
        • Oklahoma State University (OSU) - Stephenson Cancer Center
      • Tulsa, Oklahoma, Spojené státy, 74146
        • Oklahoma Cancer Specialist
    • Pennsylvania
      • Gettysburg, Pennsylvania, Spojené státy, 17325
        • Gettysburg-PCSRI
      • Langhorne, Pennsylvania, Spojené státy, 19047
        • Alliance Cancer Specialists
      • Philadelphia, Pennsylvania, Spojené státy, 19107
        • Thomas Jefferson University
      • York, Pennsylvania, Spojené státy, 17403
        • Cancer Care Associates of York - Parent
    • South Carolina
      • Greenville, South Carolina, Spojené státy, 29605
        • Prisma Health Cancer Institute
    • South Dakota
      • Sioux Falls, South Dakota, Spojené státy, 57105
        • Avera Cancer Institute
      • Sioux Falls, South Dakota, Spojené státy, 57117
        • Sanford Health
    • Tennessee
      • Dickson, Tennessee, Spojené státy, 37055
        • Sarah Cannon and HCA Research Institute
      • Franklin, Tennessee, Spojené státy, 37067
        • Sarah Cannon and HCA Research Institute
      • Gallatin, Tennessee, Spojené státy, 37066
        • Sarah Cannon and HCA Research Institute
      • Germantown, Tennessee, Spojené státy, 38138
        • West Cancer Center
      • Henderson, Tennessee, Spojené státy, 37075
        • Sarah Cannon and HCA Research Institute
      • Hermitage, Tennessee, Spojené státy, 37076
        • Sarah Cannon and HCA Research Institute
      • Lebanon, Tennessee, Spojené státy, 37090
        • Sarah Cannon and HCA Research Institute
      • Memphis, Tennessee, Spojené státy, 38120
        • Baptist Cancer Center
      • Murfreesboro, Tennessee, Spojené státy, 37129
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Spojené státy, 37203
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Spojené státy, 37205
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Spojené státy, 37207
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Spojené státy, 37211
        • Sarah Cannon and HCA Research Institute
      • Shelbyville, Tennessee, Spojené státy, 37160
        • Sarah Cannon and HCA Research Institute
      • Smyrna, Tennessee, Spojené státy, 37167
        • Sarah Cannon and HCA Research Institute
    • Texas
      • Dallas, Texas, Spojené státy, 75230
        • Mary Crowley Cancer Research
      • Dallas, Texas, Spojené státy, 75246
        • Texas Oncology - DFW
      • El Paso, Texas, Spojené státy, 79915
        • Texas Oncology
      • Houston, Texas, Spojené státy, 77030
        • MD Anderson Cancer Center
      • Houston, Texas, Spojené státy, 77030
        • Oncology Consultants
      • Irving, Texas, Spojené státy, 75063
        • Arizona Oncology Associates
      • Kingwood, Texas, Spojené státy, 77339
        • Lumi Research
      • McAllen, Texas, Spojené státy, 78503
        • Texas Oncology Central-South
      • San Antonio, Texas, Spojené státy, 78229
        • South Texas Accelerated Research Therapeutics (START)
      • Tyler, Texas, Spojené státy, 75702
        • Texas Oncology
    • Utah
      • Ogden, Utah, Spojené státy, 84405
        • Community Cancer Trials of Utah
      • Salt Lake City, Utah, Spojené státy, 84106
        • Utah Cancer Specialists
    • Virginia
      • Fairfax, Virginia, Spojené státy, 22031
        • Virginia Cancer Specialists
      • Fredericksburg, Virginia, Spojené státy, 22408
        • Hematology Oncology Associates of Fredericksburg
      • Richmond, Virginia, Spojené státy, 23235
        • Virginia Urology
    • Washington
      • Seattle, Washington, Spojené státy, 98109
        • University of Washington Cancer Consortium
      • Spokane, Washington, Spojené státy, 99216
        • Cancer Care Northwest
      • Spokane, Washington, Spojené státy, 99202
        • Spokane Urology
      • Tacoma, Washington, Spojené státy, 98405
        • Northwest Medical Specialties
    • Wisconsin
      • Appleton, Wisconsin, Spojené státy, 54911
        • ThedaCare
      • La Crosse, Wisconsin, Spojené státy, 54601
        • Gunderson Health System
      • Madison, Wisconsin, Spojené státy, 53705
        • University of Wisconsin - Carbone Cancer Center

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

12 let a starší (Dítě, Dospělý, Starší dospělý)

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Všechno

Popis

Inclusion Criteria:

  1. Patients must have a malignant solid tumor with a pathogenic inactivating TSC1 or TSC2 alteration. Genetic alterations should be identified using NGS in tumor tissue or liquid biopsy).

    • Patients will be enrolled after the central evaluation of NGS report confirms eligibility.

  2. Patients must have solid tumors that are metastatic or locally advanced where surgical resection is not an option or likely to result in severe morbidity.

  3. Patients must have received all standard therapies appropriate for their tumor type and stage of disease or, in the opinion of the Investigator, the patient would be unlikely to tolerate or derive clinically meaningful benefit from appropriate standard of care therapy, or the patient has no satisfactory alternative treatments.

  4. Patients must have 1 or more measurable target lesions by computed tomography (CT) scan or magnetic resonance imaging (MRI) (RECIST v1.1).

  5. Age: 12 years or older.

  6. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1 or Karnofsky Performance Status (KPS) ≥80 or Lansky play-performance scale for pediatric patients ≥80.

  7. Adequate liver function:

    1. Total bilirubin ≤1.5 × upper limit of normal (ULN) (unless due to Gilbert's syndrome, then ≤3 × ULN)
    2. Aspartate aminotransferase (AST) ≤2.5 × ULN (≤5 × ULN if attributable to liver metastases)

  8. Adequate renal function: creatinine clearance ≥30 mL/min, Cockcroft-Gault CCr = ((140-age) × weight[kg]) / (72 × SCr[mL/min]) × 0.85, if female

  9. Adequate hematologic parameters:

    1. Absolute neutrophil count (ANC) ≥1.0 × 109/L (growth factor support allowed)
    2. Platelet count ≥100,000/mm3 (100 × 109/L) (transfusion and/or growth factor support allowed)
    3. Hemoglobin ≥8.0 g/dL (transfusion and/or growth factor support allowed)

  10. Fasting serum triglyceride must be ≤300 mg/dL; fasting serum cholesterol must be ≤350 mg/dL.

  11. Minimum of 4 weeks since any major surgery, completion of radiation, or completion of prior systemic anticancer therapy, or at least 5 half-lives if the prior therapy is a single agent small-molecule therapeutic, and adequately recovered from the acute toxicities of any prior therapy, including neuropathy, to Grade ≤1.

  12. Male or non-pregnant and non-breastfeeding female:

    1. Females of childbearing potential must agree to use effective contraception or abstinence without interruption from 28 days prior to starting investigational product (IP) throughout 3 months after last dose of IP and have a negative serum pregnancy test (beta human chorionic gonadotropin, β-hCG) result at screening and agree to ongoing pregnancy testing during the course of the study, and after the end of study treatment. A second form of birth control is required even if she has had a tubal ligation.
    2. Male patients must agree not to donate sperm and must practice abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study and throughout 3 months after last dose of IP. A second form of birth control is required even if he has undergone a successful vasectomy.

  13. The patient or the patient's parent(s) or legal guardian(s) understand(s) and sign(s) the informed consent.

  14. Willingness and ability to comply with scheduled visits, laboratory tests, and other study procedures.

Exclusion Criteria:

  1. Prior treatment with an mTOR inhibitor, including nab-sirolimus.

  2. Severe (Grade ≥3) ongoing infection requiring parenteral or oral anti-infective treatment, either ongoing or completed ≤7 days prior to enrollment.

  3. Patients with primary brain tumors or PEComa.

  4. Patients who have any severe and/or uncontrolled medical or psychiatric conditions or other conditions that could affect their participation including:

    1. Patients with meningeal carcinomatosis, leptomeningeal carcinomatosis, spinal cord compression, untreated brain metastases or symptomatic or unstable brain metastases. Note: Patients with stable brain metastases (defined as asymptomatic or no requirement for high-dose [defined as dexamethasone 10 mg daily or higher] or increasing dose of systemic corticosteroids) and without imminent need of radiation therapy are eligible. If applicable, patients must have completed brain radiation therapy and recovered adequately from any associated toxicity and/or complications prior to eligibility assessment. For patients who have received prior radiation therapy, post-treatment MRI scan should show no increase in brain lesion size/volume.
    2. Unstable angina pectoris, symptomatic congestive heart failure (New York Heart Association, NYHA class III or IV), myocardial infarction ≤6 months prior to first study treatment, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease.
    3. Pre-existing severely impaired lung function. If a patient has a pre-existing pulmonary condition, eligible patients should have a spirometry and diffusing capacity for carbon monoxide (DLCO) that is >50% of the normal predicted value and/or O2 saturation that is >88% at rest on room air (Note: spirometry and pulmonary function tests [PFTs] not required to be performed unless clinically indicated).
    4. Nonmalignant medical illnesses that are uncontrolled or whose control may be jeopardized by the treatment with the study therapy.
    5. A history of malignancies other than the one under treatment unless the patient is disease-free for more than 5 years from diagnosis. Note, controlled non-melanoma skin cancers, carcinoma in situ of the cervix, resected incidental prostate cancer, certain low grade hematologic malignancies (eg CLL, follicular lymphoma, etc), or other adequately treated carcinoma-in-situ may be eligible, after discussion with the medical monitor.
    6. Uncontrolled hypertension (systolic blood pressure ≥160 mm-Hg and/or diastolic blood pressure ≥100 mm Hg).
    7. Patients with history of interstitial lung disease and/or pneumonitis, or pulmonary hypertension.
    8. Individuals with known human immunodeficiency virus (HIV) infection are excluded from this study as combination antiretroviral therapy could potentially result in significant pharmacokinetic interactions. In addition, these individuals are at increased risk of serious infections due to the immunosuppressive effects of mTOR inhibition.
    9. Active Hepatitis B or Hepatitis C, with detectable viral load.

  5. Regarding concomitant medications with significant CYP3A4 and P-gp interactions, discontinuation of strong inhibitors (eg, ketoconazole, itraconazole, voriconazole, erythromycin, clarithromycin, telithromycin, and others), strong inducers (eg, rifampin, rifabutin), and known CYP3A4 substrates with a narrow therapeutic window (eg, fentanyl, alfentanil, astemizole, cisapride, dihydroergotamine, pimozide, quinidine, or terfenadine) is required at least 5 half lives prior to receiving the first dose of nab-sirolimus, whichever is longer.

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: Nerandomizované
  • Intervenční model: Přiřazení jedné skupiny
  • Maskování: Žádné (otevřený štítek)

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: Arm A: Pathogenic inactivating TSC1 alterations

Patients with pathogenic inactivating TSC1 alterations.
Lék: nab-sirolimus

Prospective phase 2, open-label, multi-institutional basket trial to determine the efficacy and safety of nab-sirolimus administered by IV infusion to patients

Ostatní jména:
  • ABI-009
Experimentální: Arm B: Pathogenic inactivating TSC2 alterations

Patients with pathogenic inactivating TSC2 alterations.
Lék: nab-sirolimus

Prospective phase 2, open-label, multi-institutional basket trial to determine the efficacy and safety of nab-sirolimus administered by IV infusion to patients

Ostatní jména:
  • ABI-009

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Overall Response Rate (ORR)
Časové okno: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression , up to 30 months.

Based on CT or MRI scans from the time of study treatment initiation until disease progression, overall response rate (ORR) is defined as the proportion of patients with best overall response (BOR) of confirmed partial response (PR) or complete response (CR) in accordance with Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 as determined by independent Radiographic Review (IRR).

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression , up to 30 months.

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Duration of Response (DOR)
Časové okno: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.

DOR is defined as the number of months from the start of CR or PR (whichever response is recorded first) and subsequently confirmed to the first date of documented progressive disease (PD) or death using CT or MRI scans in accordance with RECIST v1.1.

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.

PD is defined as ≥20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.
Disease Control Rate
Časové okno: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression, up to 30 months.

Disease control rate (DCR) is defined as BOR of confirmed CR or PR (either of any duration) or SD ≥12 weeks following study treatment initiation.

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression, up to 30 months.
Time to Response
Časové okno: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.

Time to response (TTR) is defined as the time from first dose of study drug to initial measurement of CR or PR, where CR or PR is subsequently confirmed.

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.
Progression-free Survival
Časové okno: From first dose of study treatment to the date of disease progression or death

Number of months from study treatment initiation to the date of disease progression (PD) or death due to any cause PD is defined as ≥20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
From first dose of study treatment to the date of disease progression or death
Overall Survival
Časové okno: From first dose of study treatment to the date of death

Number of months from study treatment initiation to the date of death due to any cause
From first dose of study treatment to the date of death
Patient-Reported Outcome
Časové okno: D1 of every cycle up to Cycle 24

Changes from baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QOQ-C30) v3.0 scores EORTC QOQ-C30 includes functional scales (physical, role, cognitive, emotional, social), global health status, symptom scales (fatigue, pain, nausea/vomiting), and other (dyspnoea, appetite loss, insomnia, constipation/diarrhea, financial difficulties). Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); two used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score = better quality of life.
D1 of every cycle up to Cycle 24
Incidence and Severity of Treatment-emergent and Treatment-related Adverse Events (AEs)
Časové okno: Monitored continuously from signing of Informed Consent until last dose of IP (estimated up to 30 months) plus safety follow-up of 28 days after the last dose.

Incidence and severity of treatment-emergent and treatment-related AEs as assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0
Monitored continuously from signing of Informed Consent until last dose of IP (estimated up to 30 months) plus safety follow-up of 28 days after the last dose.

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

Aadi Bioscience, Inc.

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Aktuální)

10. března 2022

Primární dokončení (Aktuální)

23. prosince 2024

Dokončení studie (Aktuální)

23. prosince 2024

Termíny zápisu do studia

První předloženo

22. října 2021

První předloženo, které splnilo kritéria kontroly kvality

22. října 2021

První zveřejněno (Aktuální)

2. listopadu 2021

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

28. října 2026

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

26. října 2026

Naposledy ověřeno

1. září 2026

Více informací

Termíny související s touto studií

Klíčová slova

Další relevantní podmínky MeSH

Další identifikační čísla studie

  • TSC-007

Plán pro data jednotlivých účastníků (IPD)

Plánujete sdílet data jednotlivých účastníků (IPD)?

NE

Informace o lécích a zařízeních, studijní dokumenty

Studuje lékový produkt regulovaný americkým FDA

Ano

Studuje produkt zařízení regulovaný americkým úřadem FDA

Ne

Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .

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